Clinical Trial of EPI-743 in Patients with Leigh Syndrome [Extended Treatment Study]

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080222577

Enrollment

Registered

2014-08-06

Start date

2014-09-02

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leigh Syndrome

Interventions

investigational material(s) Generic name etc : EPI-743 INN of investigational material : - Therapeutic category code : 399 Agents affecting metabolism, n.e.c. Dosage and Administration for Investigati

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Patients with Leigh syndrome who have completed the preceding study and are willing to continue to receive EPI-743 - Patients who had no safety problems in the preceding study and are judged by the investigator or subinvestigator to be eligible for continuation of EPI-743 etc

Exclusion criteria

Exclusion criteria: - Allergy to EPI-743 or sesame oil - Allergy to vitamin E etc.

Design outcomes

Primary

Measure	Time frame
efficacy Newcastle Peadiatric Mitochondrial Disease Scale(NPMDS) (Sections 1-3) Amount of change in NPMDS(Sections 1-3) total score of each evaluation period from baseline	—

Secondary

Measure	Time frame
safety Barry-Albright Dystonia Scale Gross Motor Function Measure Need for tracheostomy Number of deaths Total number of hospitalizations Glutathione cycle biomarkers Adverse events etc. Amount of change in Barry-Albright Dystonia Scale total score of each evaluation period from baseline etc	—

Countries

Japan

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026